Studio a lungo termine su ribitol per pazienti con distrofia muscolare a cintura tipo 2I

First visit after joining

You attend the initial visit that marks the start of the study.

During this visit a complete medical history is recorded and a physical examination is performed.

Baseline functional assessments are carried out, including the nsad questionnaire, the 10mwt (10‑meter walk test), the fvc (forced vital capacity) measured while seated, the pul 2.0 test for participants who cannot walk, and the 100mtt (100‑meter timed test).

A blood sample is taken to measure serum ck levels.

Receive study medication

You are provided with ribitol granules for oral solution.

The prescribed dose is 24 g per day.

The granules are to be taken by mixing them with water and drinking the solution.

The medication is taken daily for the entire duration of the study, which may extend up to the study end date of 2029‑09‑01.

Start daily treatment

You begin the daily intake of ribitol as instructed.

The treatment continues without interruption unless a safety concern requires temporary suspension.

Regular follow‑up visits

You attend follow‑up visits at intervals defined by the study protocol.

At each visit the study team checks for any new or worsening side effects (adverse events).

The same functional assessments performed at baseline (nsad, 10mwt, fvc, pul 2.0, 100mtt) are repeated to monitor changes over time.

Blood is drawn again to reassess serum ck levels.

All findings are recorded to evaluate the long‑term safety and efficacy of the treatment.

Final assessment at study end

You complete a final visit on or before 2029‑09‑01.

The final assessment includes the same safety checks and functional tests performed throughout the study.

The results are compared with the baseline values to determine any changes in disease progression and to confirm the long‑term safety of ribitol.